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Latest News: Research

NICE Announces Appraisal Route for Nusinersen

19 January 2018 / Posted in: Research

This will be via the Single Technology Appraisal (STA) route which is not set up to assess rare disease drugs. However, there is some promising news which may see an interim Managed Access Agreement (MAA) put in place which could give more people with SMA access.

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The Updated Standards of Care Have Now Been Published

08 January 2018 / Posted in: Research

This means work can now start on writing the updated Family Guide. SMA Support UK will be working with others on this. In the meantime, you can read a summary of the ENMC workshop review of the standards.

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Invitation to parents/main carers in the UK whose children have received nuisnersen treatment to voice your views

19 December 2017 / Posted in: Information & Support, Research

Wherever you live in the UK, we would like to be able to include your views in our Patient Group Submission to the Scottish Medicines Consortium.

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Invitation to the Scottish SMA Community to voice your views

19 December 2017 / Posted in: Information & Support, Research

The Scottish Medicines Consortium (SMC) will shortly be assessing the treatment nusinersen (Spinraza) to decide whether it can be used by NHS Scotland for the treatment of SMA Types 1, 2 and 3. We need your help with this.

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Summary and comparison of results to date for nusinersen and gene therapy (Avexis-101)

15 December 2017 / Posted in: Research

Dr Alex Murphy, our Clinical Care Research Correspondent, reviews two recent articles published in the November New England Journal of Medicine.

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AveXis Announce Phase 1 Trial of AVXS-101 for SMA Type 2

15 December 2017 / Posted in: Research

The Food and Drug Administration (FDA) has granted permission to AveXis to begin a second Phase 1 trial of their gene therapy drug, AVXS-101, with SMA Type 2 patients in the United States.

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New Phase 2 Study of Repurposed Drug for SMA

06 December 2017 / Posted in: Research

Catalyst Pharmaceuticals have announced that they are to investigate the potential of a drug called Firdapse as a treatment for people with SMA Type 3.

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Update on the Scottish Medicines Consortium's Assessment of Nusinersen

30 November 2017 / Posted in: Research

We have now received a further update and clarification that the Scottish Medicines Consortium will be assessing nusinersen for "pre-symptomatic infantile-onset and later-onset 5q SMA, but not for before birth or Adult Onset SMA".

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The NICE Technology Appraisal Programme: Genetic Alliance UK's Respsonse

22 November 2017 / Posted in: Research

Genetic Alliance voices the Rare Disease Community's concerns about the proposed changes to the NICE Technology Appraisal Programme.

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Biogen SMA Community Update to SMA Europe

17 November 2017 / Posted in: Research

Biogen’s SMA Community Update to SMA Europe covers recent trial results as well as what’s happening in terms of access in Europe and worldwide.

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